Clinical Trials in China / NCT07548411
Active, not recruiting
Phase 1/2
To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A
NCT07548411 · tracked via the Priya Life Science China tracker
Condition(s) studied
Severe Hemophilia A
Investigational drug(s) / intervention(s)
GS1191-0445 injection: A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Study summary
This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A.
GS1191-0445 is an adeno-associated virus 8 (AAV8)-delivered gene therapy designed to express B-domain deleted human factor VIII (FVIII) under the regulation of a human liver-specific promoter. Following a single intravenous administration, AAV8 gene expression cassette, which transfects hepatocytes and facilitates the specific expression and secretion of FVIII into the blood.
Eligibility
Inclusion Criteria:
1. Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws:
2. Subject must be male, aged ≥18 years old at the time of signing informed consent:
3. Participants with confirmed severe hemophilia A in their pre-admission history and based on clinical laboratory examination;
4. Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment;
5. Subject has received continuous prophylactic treatment with exogenous FVIII for one year prior to enrollment or has been treated with exogenous FVIII on demand;
6. Subject has no history of hypersensitivity or allergic reactions related to the administration of FVIII agents;
7. Subject has no history of FVIII inhibitors.
8. Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent
9. Subject is willing and able to follow planned visits, treatment plans, and other study procedures.
Exclusion Criteria:
1. The subject has any hemorrhagic disorder not related to hemophilia A,
2. Abnormal liver function test results of subjects during screening.
3. Abnormal laboratory examination of subjects during screening
4. The subject has acute or chronic hepatitis B virus (HBV) infection or chronic hepatitis C virus (HCV) infection; Or are receiving antiviral treatment for hepatitis B and C;
5. Active systemic immune disease.
Primary outcome measure(s)
- Number of participants with Adverse Events (AE) as assessed by CTCAE v5.0, including Adverse Event of Special Interests (AESI) and Serious Adverse Events (SAE); — Five years after infusion
- Number of Participants with Thrombosis Risk: In subjects with >150% FⅧ:C post-GS1191-0445 infusion, VTE risk will be assessed via Caprini model, coagulation function, D-dimer, FDP, and TAT; — Five years after infusion
- The shedding of GS1191-0445 viral vector: Viral vector titers in serum, saliva, urine, semen and fecal will be monitored; — Five years after infusion
- Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein; — Five years after infusion
- FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein; — Five years after infusion
- The number of dose-limiting toxicity (DLT) events will be determined by the Safety Review Committee (SRC), at least 12 weeks after GS1191-0445 infusion. — Five years after infusion
- The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis; — Five years after infusion
- Changes for vital signs: Includes sitting blood pressure (mmHg), respiratory rate (breaths/min), body temperature (°C), and pulse rate (beats/min); — Five years after infusion
- Changes for physical examination: Includes skin, mucous membranes, lymph nodes, head and neck, chest (heart, lungs), abdomen, muscles, nervous system, spine/extremities; — Five years after infusion
- The immunogenicity of AAV capsid protein: Collection of Peripheral Blood Mononuclear Cell (PBMC) and serum samples for vector shedding detection; — Five years after infusion
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Union Hospital, Tongji Medical College, Huazhong University of Science and Technology | Wuhan | Hubei | |
| Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College | Tianjin | Tianjin Municipality |
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07548411 on ClinicalTrials.gov ↗ ← All trials in China